The joint monothematic conference of the Italian Society of Pharmacology (SIF), entitled “Shaping the Future of Pediatric Health: Innovative Research Approaches for Rare and Metabolic Diseases in Children”, took place on 23–24 February 2026 at the Palazzo Ateneo of the University of Bari Aldo Moro. The event brought together leading national and international experts in paediatric pharmacology and rare diseases.
Organised by the SIF Working Groups on “Paediatric Pharmacology” and “Rare Diseases and Orphan Drugs – SIF4RARE”, the meeting provided an important opportunity for multidisciplinary exchange on key challenges in the development of innovative therapies for rare and metabolic diseases in children. Discussions focused on early diagnosis, drug safety, the use of real-world evidence, and the growing role of artificial intelligence in research and development processes.
The Foundation actively contributed to the scientific programme, supporting discussions on some of the most strategic issues shaping the future of pharmacological research in rare diseases. In particular, Viviana Giannuzzi, our head of Research and Innovation Department, was invited to join the Scientific Committee of the congress, and also chaired the keynote lecture session entitled “Shift to early diagnosis of rare paediatric metabolic conditions: Impact on care, treatment and drug development”. Moreover, researchers from the Foundation contributed to both oral and poster presentations: Silvia Torretta presented an oral communication entitled “Individualised treatments for rare and ultra-rare diseases: ethics and regulatory insights from a first ERDERA multi-stakeholders consensus meeting”, providing an overview of ethics and regulatory challenges related to the clinical development of individualised antisense oligonucleotide (ASO) therapies; Maria Luisa Dalessandro presented a poster titled: “An easy-to-use flow chart to classify your clinical study”, an output from the PNRR-funded BETTER project, which introduces an intuitive tool for researchers to classify clinical studies, in line with the applicable regulatory requirements and ethics provisions. The poster from Lucia Ruggieri and Sabina Sblano presented the expected impact of the Pharma Legislation revision on research and availability of paediatric medicines, outlining the contribution of the European Paediatric Translational Research infrastructure (Poster title: Defending paediatric medicines in Europe: EPTRI’s strategic contribution and actions in shaping the new pharmaceutical legislation).
The participation of the Foundation reflects its ongoing commitment to promoting scientific research and fostering dialogue among healthcare stakeholders, with the aim of advancing the development of safe, effective and accessible therapies for the most vulnerable patients.
